Long-term AGAMREE data indicates continued effectiveness with clinically meaningful safety advantages over classic corticosteroids

Santhera Pharmaceuticals announced results from the ongoing Guardian study in Duchenne, and showed that AGAMREE had comparable effectiveness compared to historical cohorts taking prednisone or deflazacort, based on time to loss of ambulation.  Moreover, individuals taking AGAMREE had fewer vertebra fractures and less incidence of cataracts compared to those taking deflazacort.  Individuals taking AGAMREE also exhibited less growth stunting compared to classic corticosteroids, with an average height advantage of 12 cm after 5 years.  

CureDuchenne provided early funding to the development of AGAMREE, which is available in the US via Catalyst Pharmaceuticals.